| 英語名 | 研究コード | 研究開発ステージ | 会社 | 適応症 | 臨床試験 |
|---|---|---|---|---|---|
| Human apotransferrin (Sanquin Blood Supply Foundation) | Phase 3 Clinical | Sanquin Plasma Products Bv | beta-Thalassemia; Iron Metabolism Disorders | 詳細 | |
| Delpacibart braxlosiran | AOC1020 | Phase 3 Clinical | Avidity Biosciences Inc | Foot-and-Mouth Disease; Muscular Dystrophies; Muscular Dystrophy, Facioscapulohumeral | 詳細 |
| Delpacibart eteddesiran | AOC-1001; AOC 1001 | Phase 3 Clinical | Avidity Biosciences Inc | Genetic Diseases, Inborn; Heredodegenerative Disorders, Nervous System; Muscular Atrophy; Muscular Diseases; Muscular Disorders, Atrophic; Muscular Dystrophies; Musculoskeletal Diseases; Myotonia; Myotonic Disorders; Myotonic Dystrophy; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Diseases | 詳細 |
| Trontinemab | RO-7126209; RG-6102 | Phase 3 Clinical | F. Hoffmann-La Roche Ltd | Alzheimer Disease | 詳細 |
| CX-2029 | ABBV-2029; ABBV-CX-2029; CX-2029 | Phase 2 Clinical | Abbvie Inc, Cytomx Therapeutics Inc | Carcinoma, Non-Small-Cell Lung; Esophageal Neoplasms; Head and Neck Neoplasms; Lymphoma, Large B-Cell, Diffuse; Solid tumours | 詳細 |
| Zeleciment rostudirsen | DYNE251 | Phase 2 Clinical | Dyne Therapeutics Inc | Muscular Dystrophy, Duchenne | 詳細 |
| AOC-1044 | AOC-1044; AOC 1044 | Phase 2 Clinical | Avidity Biosciences Inc | Muscular Dystrophy, Duchenne | 詳細 |
| JR-446 | JR-446 | Phase 2 Clinical | Jcr Pharmaceuticals Co Ltd, Medipal Holdings Corp | Mucopolysaccharidosis III | 詳細 |
| JR-441 | JR-441 | Phase 2 Clinical | Jcr Pharmaceuticals Co Ltd | Mucopolysaccharidosis III | 詳細 |
| Lepunafusp alfa | JR-171 | Phase 2 Clinical | Jcr Pharmaceuticals Co Ltd | Mucopolysaccharidosis I | 詳細 |
| PPMX-T003 | PPMX-T003 | Phase 2 Clinical | Perseus Proteomics Inc | Leukemia; Polycythemia Vera | 詳細 |
| INA-03 | INA03 | Phase 1 Clinical | Inatherys | Leukemia; Leukemia, Myeloid, Acute; Precursor Cell Lymphoblastic Leukemia-Lymphoma | 詳細 |
| PRX012-TfR | PRX012-TfR | Phase 1 Clinical | Prothena | Alzheimer Disease | 詳細 |
| KK-2260 | KK2260; KK-2260 | Phase 1 Clinical | Kyowa Kirin Co Ltd | Esophageal Neoplasms; Solid tumours; Squamous Cell Carcinoma of Head and Neck | 詳細 |
| ABX-1100 | ABX-1100 | Phase 1 Clinical | Aro Biotherapeutics Co | Glycogen Storage Disease Type II | 詳細 |
| 英語名 | 研究コード | 研究開発ステージ | 会社 | オリジナル製品名 | 最初に承認をした国 | 最初の適応症 | 最初に承認を受けた会社 | 承認された日付 | 適応症 | 臨床試験 |
|---|---|---|---|---|---|---|---|---|---|---|
| Pabinafusp alfa | JR-141 | Approved | Jcr Pharmaceuticals Co Ltd | Japan | Mucopolysaccharidosis II | Jcr Pharmaceuticals Co Ltd | 2021-03-23 | Mucopolysaccharidosis II | 詳細 |





